A Clinical Trial of Elsunersen in Pediatric SCN2A-DEE to Assess Efficacy and Safety (NCT07019922) | Clinical Trial Compass
RecruitingPhase 3
A Clinical Trial of Elsunersen in Pediatric SCN2A-DEE to Assess Efficacy and Safety
United States50 participantsStarted 2025-08-13
Plain-language summary
A Randomized, Multi-Center, Double-Blind, Sham-Procedure-Controlled Clinical Trial to Investigate the Efficacy and Safety of Elsunersen in Pediatric Participants with Early Onset SCN2A Developmental and Epileptic Encephalopathy
Who can participate
Age range1 Day – 18 Years
SexALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion Criteria:
* Has a documented Gain of Function SCN2A variant confirmed through genetic testing.
* Has onset of seizures prior to 3 months of age.
* Seizure frequency of 4 or more countable motor seizures per 28-day during the Baseline Observation Period.
Exclusion Criteria:
* Has any clinically significant or known pathogenic genetic variant other than in the SCN2A gene, or a genetic variant that may explain or contribute to the participant's epilepsy and/or developmental disorder.
* Has bone, spine (eg, kyphosis, scoliosis), bleeding, or other disorder.
* Has received any experimental or investigational drug, device, or other therapy within 30 days or 5 half-lives (whichever is longer) prior to Screening, including any prior use of gene therapy.
* Is currently pregnant or breastfeeding or is planning to become pregnant during the clinical trial.
What they're measuring
1
To assess the efficacy of elsunersen on seizure frequency in participants with early-onset SCN2A DEE